JP2020511155A5 - - Google Patents

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JP2020511155A5
JP2020511155A5 JP2019552274A JP2019552274A JP2020511155A5 JP 2020511155 A5 JP2020511155 A5 JP 2020511155A5 JP 2019552274 A JP2019552274 A JP 2019552274A JP 2019552274 A JP2019552274 A JP 2019552274A JP 2020511155 A5 JP2020511155 A5 JP 2020511155A5
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JP
Japan
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compound
pharmaceutically acceptable
wing segment
compound according
modified oligonucleotide
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JP2019552274A
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Japanese (ja)
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JP2020511155A (ja
JP7022143B2 (ja
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Priority claimed from PCT/US2018/023936 external-priority patent/WO2018175839A1/en
Publication of JP2020511155A publication Critical patent/JP2020511155A/ja
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Priority to JP2021208929A priority Critical patent/JP7289347B2/ja
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Publication of JP7022143B2 publication Critical patent/JP7022143B2/ja
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JP2019552274A 2017-03-24 2018-03-23 Pcsk9発現のモジュレーター Active JP7022143B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021208929A JP7289347B2 (ja) 2017-03-24 2021-12-23 Pcsk9発現のモジュレーター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762476051P 2017-03-24 2017-03-24
US62/476,051 2017-03-24
PCT/US2018/023936 WO2018175839A1 (en) 2017-03-24 2018-03-23 Modulators of pcsk9 expression

Related Child Applications (1)

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JP2021208929A Division JP7289347B2 (ja) 2017-03-24 2021-12-23 Pcsk9発現のモジュレーター

Publications (3)

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JP2020511155A JP2020511155A (ja) 2020-04-16
JP2020511155A5 true JP2020511155A5 (es) 2021-12-09
JP7022143B2 JP7022143B2 (ja) 2022-02-17

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JP2019552274A Active JP7022143B2 (ja) 2017-03-24 2018-03-23 Pcsk9発現のモジュレーター
JP2021208929A Active JP7289347B2 (ja) 2017-03-24 2021-12-23 Pcsk9発現のモジュレーター
JP2023088955A Pending JP2023109989A (ja) 2017-03-24 2023-05-30 Pcsk9発現のモジュレーター

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JP2021208929A Active JP7289347B2 (ja) 2017-03-24 2021-12-23 Pcsk9発現のモジュレーター
JP2023088955A Pending JP2023109989A (ja) 2017-03-24 2023-05-30 Pcsk9発現のモジュレーター

Country Status (27)

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US (4) US10517953B2 (es)
EP (2) EP4309732A3 (es)
JP (3) JP7022143B2 (es)
KR (1) KR102645714B1 (es)
CN (1) CN110446784A (es)
AR (1) AR113214A1 (es)
AU (2) AU2018237306B2 (es)
BR (1) BR112019015041A2 (es)
CA (1) CA3056129A1 (es)
CL (2) CL2019002574A1 (es)
CO (1) CO2019011265A2 (es)
CR (1) CR20190471A (es)
DO (1) DOP2019000232A (es)
EA (1) EA201992170A1 (es)
EC (1) ECSP19072372A (es)
IL (2) IL269273A (es)
JO (1) JOP20190215A1 (es)
MX (1) MX2019010699A (es)
MY (1) MY194227A (es)
NI (1) NI201900096A (es)
NZ (1) NZ758177A (es)
PE (1) PE20191484A1 (es)
PH (1) PH12019502174A1 (es)
SG (1) SG11201908393YA (es)
TW (2) TW202325310A (es)
UA (1) UA127390C2 (es)
WO (1) WO2018175839A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018216785A1 (ja) * 2017-05-26 2018-11-29 国立研究開発法人国立循環器病研究センター Pcsk9を標的としたアンチセンス核酸
US20230357768A1 (en) * 2020-09-17 2023-11-09 Astrazeneca Ab Pharmaceutical compositions comprising an antisense oligonucleotide for oral administration
US20230355653A1 (en) 2020-09-17 2023-11-09 Astrazeneca Ab Pcsk9 inhibitors and methods of treatment using same
WO2022089486A1 (zh) * 2020-10-28 2022-05-05 江苏柯菲平医药股份有限公司 抑制PCSK9基因表达的siRNA及其修饰物与应用
BR112023021766A2 (pt) 2021-04-22 2024-01-23 Civi Biopharma Inc Dispensação oral de oligonucleotídeo

Family Cites Families (143)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US4751219A (en) 1985-02-05 1988-06-14 Nederlandse Centrale Organisatie Voor Toegepast-Natuur-Wetenschappelijk Onderzoek Synthetic glycolipides, a process for the preparation thereof and several uses for these synthetic glycolipides
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
ATE113059T1 (de) 1987-06-24 1994-11-15 Florey Howard Inst Nukleosid-derivate.
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5859221A (en) 1990-01-11 1999-01-12 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5948903A (en) 1991-01-11 1999-09-07 Isis Pharmaceuticals, Inc. Synthesis of 3-deazapurines
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
AU3222793A (en) 1991-11-26 1993-06-28 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US6172045B1 (en) 1994-12-07 2001-01-09 Neorx Corporation Cluster clearing agents
US6908903B1 (en) 1994-12-07 2005-06-21 Aletheon Pharmaceuticals, Inc. Cluster clearing agents
CN1120707C (zh) 1995-11-22 2003-09-10 约翰斯·霍普金斯大学 增强生物分子的细胞摄取的配体
US20030119724A1 (en) 1995-11-22 2003-06-26 Ts`O Paul O.P. Ligands to enhance cellular uptake of biomolecules
CN1231675A (zh) 1996-09-26 1999-10-13 味之素株式会社 修饰的生理活性蛋白及含有该蛋白的药物组合物
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
KR20070118315A (ko) 1999-04-21 2007-12-14 와이어쓰 폴리뉴클레오티드 서열의 기능을 억제하기 위한 조성물
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6383812B1 (en) 1999-05-28 2002-05-07 Academia Sinica Anti liver disease drug R-YEEE and method of synthesizing branched galactose-terminal glycoproteins
US8541548B2 (en) 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US8137695B2 (en) 2006-08-18 2012-03-20 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US6906182B2 (en) 2000-12-01 2005-06-14 Cell Works Therapeutics, Inc. Conjugates of glycosylated/galactosylated peptide, bifunctional linker, and nucleotidic monomers/polymers, and related compositions and method of use
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US20070173473A1 (en) * 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
US20030175906A1 (en) 2001-07-03 2003-09-18 Muthiah Manoharan Nuclease resistant chimeric oligonucleotides
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US20100240730A1 (en) 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
EP1543019A2 (en) 2002-09-11 2005-06-22 Santaris Pharma A/S Modified pna molecules
EP2957568B1 (en) 2002-11-05 2016-12-21 Ionis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US6673661B1 (en) 2002-12-20 2004-01-06 Taiwan Semiconductor Manufacturing Co., Ltd. Self-aligned method for forming dual gate thin film transistor (TFT) device
EP2669377A3 (en) 2003-04-17 2015-10-14 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
JPWO2004101619A1 (ja) 2003-05-15 2006-10-26 塩野義製薬株式会社 機能的糖ペプチドの合理的設計および合成
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
CA2538252C (en) 2003-09-18 2014-02-25 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
WO2006020768A2 (en) 2004-08-10 2006-02-23 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides
US20090203132A1 (en) 2004-09-09 2009-08-13 Swayze Eric E Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
WO2006078217A1 (en) 2005-01-24 2006-07-27 Avaris Ab COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
CN102796147A (zh) * 2005-10-04 2012-11-28 阿尔卡米亚有限公司 化合物库
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
WO2007143317A2 (en) * 2006-05-05 2007-12-13 Isis Pharmaceuticals, Inc Compounds and methods for modulating expression of crp
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
JP5441688B2 (ja) 2006-05-11 2014-03-12 アイシス ファーマシューティカルズ, インコーポレーテッド 5’修飾二環式核酸類似体
US7605251B2 (en) * 2006-05-11 2009-10-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
US8658211B2 (en) 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
DK2410054T4 (da) 2006-10-18 2020-02-10 Ionis Pharmaceuticals Inc Antisenseforbindelser
US8084437B2 (en) * 2006-11-27 2011-12-27 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
WO2008098788A2 (en) 2007-02-16 2008-08-21 Ktb Tumorforschungsgesellschaft Mbh Receptor and antigen targeted prodrug
WO2008109369A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
AU2008242583B2 (en) 2007-04-23 2013-10-10 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
ES2388590T3 (es) 2007-05-30 2012-10-16 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos con puente aminometileno N-sustituido.
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009023855A2 (en) 2007-08-15 2009-02-19 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
US20090247608A1 (en) 2007-12-04 2009-10-01 Alnylam Pharmaceuticals, Inc. Targeting Lipids
AU2009241591A1 (en) 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of DSRNA targeting the PCSK9 gene
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
US20110065644A1 (en) * 2008-03-09 2011-03-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
WO2009120878A2 (en) 2008-03-26 2009-10-01 Alnylam Pharmaceuticals, Inc. Non-natural ribonucleotides, and methods of use thereof
CA2721183C (en) 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2009148605A2 (en) 2008-06-04 2009-12-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
CA2737661C (en) 2008-09-23 2019-08-20 Alnylam Pharmaceuticals, Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
US8501805B2 (en) 2008-09-24 2013-08-06 Isis Pharmaceuticals, Inc. Substituted alpha-L-bicyclic nucleosides
EP4241767A3 (en) 2008-11-10 2023-11-01 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
CA2751342C (en) 2009-01-29 2019-05-07 Alnylam Pharmaceuticals, Inc. Lipid formulations comprising cationic lipid and a targeting lipid comprising n-acetyl galactosamine for delivery of nucleic acid
NZ711583A (en) 2009-05-05 2017-03-31 Arbutus Biopharma Corp Lipid compositions
CA2764609C (en) 2009-06-10 2018-10-02 Alnylam Pharmaceuticals, Inc. Improved cationic lipid of formula i
KR20120050429A (ko) 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Pcsk9 유전자를 표적으로 하는 지질 제형된 dsrna
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
TWI458493B (zh) 2009-09-25 2014-11-01 Iner Aec Executive Yuan 新穎肝標靶藥劑與合成方法
TWI391144B (zh) 2009-10-26 2013-04-01 Iner Aec Executive Yuan 一種定量肝殘餘功能的檢驗方法與其新穎肝受體造影檢驗藥劑
TWI388338B (zh) 2009-10-26 2013-03-11 Iner Aec Executive Yuan 對聚合醣鏈進行放射標誌以作為肝受體造影劑之方法
WO2011072290A2 (en) 2009-12-11 2011-06-16 The Regents Of The University Of Michigan Targeted dendrimer-drug conjugates
WO2011100131A2 (en) 2010-01-28 2011-08-18 Alnylam Pharmacuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
SG183374A1 (en) 2010-02-24 2012-09-27 Arrowhead Res Corp Compositions for targeted delivery of sirna
WO2011120053A1 (en) 2010-03-26 2011-09-29 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
US20130109817A1 (en) 2010-03-26 2013-05-02 Mersana Therapeutics, Inc. Modified Polymers for Delivery of Polynucleotides, Method of Manufacture, and Methods of Use Thereof
US9102938B2 (en) 2010-04-01 2015-08-11 Alnylam Pharmaceuticals, Inc. 2′ and 5′ modified monomers and oligonucleotides
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
US20130236968A1 (en) 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
JP2013541334A (ja) 2010-09-15 2013-11-14 アルニラム ファーマスーティカルズ インコーポレイテッド 修飾されたiRNA剤
EP2640400A4 (en) 2010-11-19 2016-01-20 Sirna Therapeutics Inc POLYMERIC POLYMERS (AMIDE) FOR THE ADMINISTRATION OF OLIGONUCLEOTIDES
EP2652134B1 (en) 2010-12-17 2017-03-01 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA3131967A1 (en) 2010-12-29 2012-07-05 F. Hoffman-La Roche Ag Small molecule conjugates for intracellular delivery of nucleic acids
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
JP6236385B2 (ja) 2011-06-21 2017-11-22 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. アポリポタンパク質c−iii(apoc3)の発現を阻害するための組成物及び方法
US8932572B2 (en) 2011-08-26 2015-01-13 Arrowhead Madison Inc. Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EP2751270B1 (en) 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
IL284530B (en) 2011-11-18 2022-07-01 Alnylam Pharmaceuticals Inc rnai factors, preparations and methods of using them for the treatment of transthyretin-related diseases
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP3919620A1 (en) 2012-05-02 2021-12-08 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
AR090906A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos
EP2906699A4 (en) 2012-10-11 2016-06-08 Ionis Pharmaceuticals Inc OLIGOMER COMPOUNDS WITH BICYCLIC NUCLEOSIDES AND USES THEREOF
EP4083209A1 (en) 2012-12-05 2022-11-02 Alnylam Pharmaceuticals, Inc. Pcsk9 irna compositions and methods of use thereof
US9450467B2 (en) 2013-03-14 2016-09-20 Summin Holding Company, Llc Submersible pump
RU2019110030A (ru) * 2013-05-01 2019-05-06 Ионис Фармасьютикалз, Инк. Композиции и способы
CN105358692B (zh) * 2013-06-27 2020-08-21 罗氏创新中心哥本哈根有限公司 靶向pcsk9的反义寡聚体和缀合物
CA2928349A1 (en) * 2013-11-14 2015-05-21 Roche Innovation Center Copenhagen A/S Apob antisense conjugate compounds
WO2015106128A2 (en) 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
CN106232804B (zh) * 2014-05-01 2019-10-25 Ionis制药公司 用于调节补体因子b表达的组合物和方法
US20170145424A1 (en) 2014-06-06 2017-05-25 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
JOP20190112A1 (ar) 2016-11-14 2019-05-14 Amgen Inc علاجات مدمجة لتصلب الشرايين، شاملة مرض قلبي وعائي تصلبي
CN110582570A (zh) * 2017-02-22 2019-12-17 克里斯珀医疗股份公司 用于治疗前蛋白转化酶枯草杆菌蛋白酶/Kexin 9型(PCSK9)相关的病症的组合物和方法
WO2018216785A1 (ja) 2017-05-26 2018-11-29 国立研究開発法人国立循環器病研究センター Pcsk9を標的としたアンチセンス核酸
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

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